Kang Shuo, Zhao Shan, Wang Xiaohui, Pan Zhenhua
Medical Insurance Office, The Second Hospital of Hebei Medical University, Shijiazhuang, Hebei Province, People's Republic of China.
Department of Oncology, The Second Hospital of Hebei Medical University, Shijiazhuang, Hebei Province, People's Republic of China.
PLoS One. 2025 Jul 24;20(7):e0328730. doi: 10.1371/journal.pone.0328730. eCollection 2025.
With or without biomarker-directed toripalimab plus chemotherapy could bring significant clinical benefit and acceptable safety profile compared with chemotherapy as first-line treatment for patients with extensive-stage small-cell lung-cancer (ES-SCLC) were demonstrated in EXTENTORCH trial. However, its cost-effective remains uncleared.
The current analysis aimed to evaluate the economic value of intratumor heterogeneity (ITH) testing directed toripalimab plus chemotherapy as first-line treatment for patients with ES-SCLC from the Chinese health-care system perspective.
A mathematical decision model-based cost-effectiveness analysis.
A pharmacoeconomic decision model was developed to simulate 3-week patients transition in 20-year time horizon to access the cost-effectiveness of three competing first-line treatments among ITH-testing directed toripalimab plus chemotherapy, toripalimab plus chemotherapy, and chemotherapy alone. Survival data were obtained from EXTENTORCH trial, cost and utility values were gathered from the dataset and published studies, annual discount rate of 5% was used for cost and utility values. Total cost, life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) were the model outputs. One-way and probabilistic sensitivity analyses were conducted to estimate the robustness of the model results.
In base-case analysis, compared with toripalimab plus chemotherapy and chemotherapy alone, ITH-testing directed therapy could bring additional 0.14 QALYs and 0.29 QALYs, with marginal costs of $3750.75 and $7778.18, resulting in the ICER of $27,353.27/QALY and $26,461.46/QALY, respectively, which lower than the Chinese willingness-to-pay (WTP) threshold. Sensitivity analyses demonstrated the model results were robust, probabilistic sensitivity analyses showed the probability of ITH-testing directed therapy could be considered cost-effective was 61%.
ITH-testing directed treatment was likely to be the most cost-effective first-line option compared with toripalimab plus chemotherapy and chemotherapy alone for patients with previously untreated ES-SCLC from the Chinese health-care system perspective.
EXTENTORCH试验表明,对于广泛期小细胞肺癌(ES-SCLC)患者,无论有无生物标志物指导,托瑞帕利单抗联合化疗作为一线治疗与单纯化疗相比,均可带来显著的临床获益和可接受的安全性。然而,其成本效益仍不明确。
本分析旨在从中国医疗保健系统的角度评估肿瘤内异质性(ITH)检测指导的托瑞帕利单抗联合化疗作为ES-SCLC患者一线治疗的经济价值。
基于数学决策模型的成本效益分析。
开发了一个药物经济学决策模型,以模拟20年时间范围内3周的患者转变情况,以评估ITH检测指导的托瑞帕利单抗联合化疗、托瑞帕利单抗联合化疗和单纯化疗这三种一线治疗方案的成本效益。生存数据来自EXTENTORCH试验,成本和效用值从数据集和已发表的研究中收集,成本和效用值采用5%的年贴现率。总成本、生命年(LYs)、质量调整生命年(QALYs)和增量成本效益比(ICER)是模型输出。进行单因素和概率敏感性分析以评估模型结果的稳健性。
在基础案例分析中,与托瑞帕利单抗联合化疗和单纯化疗相比,ITH检测指导的治疗可带来额外的0.14个QALY和0.29个QALY,边际成本分别为3750.75美元和7778.18美元,ICER分别为27353.27美元/QALY和26461.46美元/QALY,均低于中国的支付意愿(WTP)阈值。敏感性分析表明模型结果稳健,概率敏感性分析显示ITH检测指导的治疗被认为具有成本效益的概率为61%。
从中国医疗保健系统的角度来看,对于先前未治疗的ES-SCLC患者,ITH检测指导的治疗可能是与托瑞帕利单抗联合化疗和单纯化疗相比最具成本效益的一线选择。